Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Mucopolysaccharidosis Type III A Clinical Trial

Official title:

Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02053064
• Other study ID #: P2-SAF-301
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: January 16, 2014
• Last updated: June 19, 2017
• Start date: May 2013
• Est. completion date: June 2017

Study information

• Verified date: June 2017
• Source: LYSOGENE
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: June 2017
• Est. primary completion date: May 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients having completed the previous phase I/II study (P1-SAF-301),

• Family understanding the follow-up procedures and the informed consent,

• Patient's parents having signed the informed consent form.

Exclusion Criteria:

• Participation in any other clinical trial.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis Type III A
• Sanfilippo Disease Type A

Intervention

Genetic:
• SAF-301

Locations

Country	Name	City	State
France	Hôpitaux Universitaires Paris Sud (Bicêtre)	Le Kremlin-Bicêtre

Sponsors (1)

Lead Sponsor	Collaborator
LYSOGENE

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Checking of adverse events		until 60 months after SAF-301 administration
Secondary	Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments		5 years after the injection of SAF-301
Secondary	Information on changes in potential biomarkers of the disease		5 years after the injection of SAF-301
Secondary	Information to further evaluation of immune response		5 years after the injection of SAF-301